FDA Issues Final Guidance on Potency Assurance for Cell and Gene Therapies
The U.S. Food and Drug Administration (FDA) on November 2 issued final guidance for manufacturers of cellular and gene therapy (CGT) products, recommending a comprehensive, science- and risk-based Potency Assurance Strategy to test and verify — before a product reaches a patient — that every batch meets a defined standard of potency to do what the product is supposed to do therapeutically.
The final guidance, "Potency Assurance for Cellular and Gene Therapy Products," replaces the FDA's 2011 potency testing guidance for CGT products and applies to treatments for serious and often life-threatening conditions such as cancers and rare diseases. By law, manufacturers are expected to verify that their products work as intended, from the time of approval through post-approval monitoring.
"Patients who receive cellular and gene therapies are often battling serious or life-threatening conditions, such as cancers and rare diseases, and they deserve the assurance that the product administered to them will perform as intended," said Karim Mikhail, Director of the Center for Biologics Evaluation and Research. "This guidance reflects the FDA's commitment to ensuring America remains the best place in the world to innovate — and that innovation is matched by the highest standards of product quality and patient safety."
The guidance offers flexible, risk-based recommendations covering every stage from early-phase investigational new drug (IND) submissions through biologics license application (BLA) approval and continued post-market verification, including controls for variability in raw materials, testing during manufacturing, and lab tests confirming each batch's biological activity. It also addresses products with short shelf lives, viral vector gene therapies and patient-specific therapies. The FDA will host a webinar on December 10, 2026, to help stakeholders implement the guidance.
(Source: U.S. Food and Drug Administration)
Source: GSM · USA (original Chinese edition)
Photo: 图：美国食品药品监督管理局（FDA）位于马里兰州 Silver Spring 的 White Oak 园区总部大楼。FDA 官方图，公共领域，经 Wikimedia Commons。 / Photo: FDA headquarters buildings at the White Oak campus in Silver Spring, Maryland. U.S. Food and Drug Administration, public domain, via Wikimedia Commons.
